PF-02341066 trial moving to Phase III

David, Peter and I drove to Boston early this morning for my routine trial appointment. The trial team has decided that I only need to come to the hospital once a month now.   My scan regimen has changed slightly as well. Although I must still have scans every two months, it won’t be necessary to get the abdominal scan each time. That means no more barium milkshakes for the moment. YES for small victories.   My oncologist, Dr. Shaw, just returned from the 13th WCLC (World Conference on Lung Cancer) in San Francisco this past weekend. At the meeting, Dr. Shaw presented updated data on the efficacy of the PF-02341066 trial. The results continue to be very promising and continue to generate excitement in the field of thoracic oncology.
The trial will soon enter Phase III. Prior to FDA approval of a drug, a controlled and randomized trial must be conducted.  It will be at multiple locations around the globe and will enroll many more people.  All those enrolling in Phase III must have the ALK mutation as well as only one failed first line therapy.  The fact that it is randomized means that some subjects will receive standardized chemo rather than the trial drug. However, should they suffer adverse effects from the standard chemo, or have progression of disease at their first scan at six weeks, they will be permitted to switch to the trial drug.  A phase II of the trial will be running simultaneously (the primary goal of Phase I is to assess for safety and dose escalation, Phase II is efficacy of the drug). Those subjects who possess the ALK mutation, but have also had more than one failed first line therapy, may enroll in a Phase II of the trial. So–bottom line–you need to have the mutation.

It was also fun to see images of my CT scans that were used as slides at the presentation. She also showed us a chart of subject response and it was interesting to see the hard data. Although my own response was characterized by my oncologist and the radiologist as “an almost total response”, statistically it is approx. a 70%  PR, or partial response.

It is beyond me how they determine such things (although Dr. Shaw explained that the methodology is to assess a two dimensional shrinkage of tumor as shown by scans). My CT scan could hardly look much better. However, I know that we are continuing to watch an area that represents either scarring or lung cancer. Only the statistical percentage is surprising, and I learned early in this journey to not pay too much attention to numbers. I just know how good I feel and that I continue to wake up every morning.  Wasn’t it Woody Allen who said that “80% of success is showing up”?
We also talked more about how the trial drug actually works.  The mutation that I have acquired (it is not inherited and so has been caused by exposure to one or more carcinogens) means that my DNA has been permanently altered, and altered in such a way that it makes cancerous cells.  The altered gene has become an oncogene:  a gene that contributes to the conversion of a normal cell into a cancerous one.  By locating where that mutation was (on the ALK gene), researchers were able to pinpoint a target.  The trial drug (PF-02341066) is a targeted therapy.   It is referred to as cancer growth blocker.  Growth factors trigger the cancer cells to divide and grow, and cancer cells are often very sensitive to them.  If these growth factors can be blocked, it is possible to stop growth and division of the cancer cells.

PF-02341066 is a Tyrosine kinase inhibitor.  This refers to the type of chemical that it blocks:  in this case, enzymes called tyrosine kinases.  These enzymes attach to a receptor on a cancerous cell and signal it to divide.  The Tyroisne kinase inhibitor stops that signal.  In layman’s terms:  my cancer has not been cured.  It has been stalled, the growth and division of cancerous cells halted, possibly indefinitely.

Realistically, it is unknown how long this drug will be effective for.  When and if it stops working, the cancer will begin to grow again.  I hope for two things: that the cancer will be held at bay for a long time, and that in the meantime, other treatments are developed.  This is the way it is with cancer.  We, all of us with cancer, are truly at the frontier.  There is so much yet to discover, and I am so grateful to those who are dedicating their life work to this end. My very existence depends on it.

11 responses to “PF-02341066 trial moving to Phase III

  1. I think the part where you talked about not thinking too much about the numbers is very important. The statistics and numbers tend to be very scary and it is so easy to just get overwhelmed.

    It was also very informative/interesting to have you write about how the drug actually works. I had no idea how it did it… might as well have been waving a wand and saying SHAZAM! So thank you for spelling it out!!!

    love you

    ❤ jemesii

  2. I had to figure out how the drug worked before I could write that–I’m glad I understand now too. Love, Mom

  3. Hi:
    In Match Point Woody Allen movie the random, the good luck, allows Chris to stay free of his difficult situation, when the ball hits the net is better to be lucky than good.
    We are lucky and survive for another game, Kevin was young and good and unfortunately unlucky.
    By choices we can help our luck. I left Buenos Aires in 1969, I was told that I was crazy, god was Argentinean, after the dirty war and Falkland’s war the country is bankrupt. I learned English in Vermont in 4 months, lucky got a free new car to deliver with other students from Niagara Falls to L. A. visiting Grand Canyon and Vegas. I got a programmer/analyst job in San Francisco and bought an old car cash, lucky me. Unlucky me could not get a green card. Lucky I was accepted for Australia and Canada and after working 25 years in Toronto I took early retirement in 1995, age 56, I was told that I was crazy again. My house was paid, my two daughters got university education and work was a 4 letter word, I choose freedom and for 10 years relaxed and sailed in long beautiful trips. Like you I went to Boston, but in the schooner Empire Sandy from Bahamas. Unlucky in 2005 with lung cancer diagnosis. Like you I never smoked, may have the EML4-ALK mutation, my wife was depressed and was treated well. I accepted my luck and luckily found a free for Canadians great Cancer center in Princess Margaret Hospital, Toronto.
    I see that you are lucky to have a lovely daughter, me to.
    Like you I like vacations, after chemo in the fall I’m planning to go to California and show my English wife San Francisco, Yosemite, Lake Tahoe and cruise back Pacific coast and Panama canal, Caribbean to Florida.
    Keep playing.

  4. Loving your story…best of luck going forward. You are a beacon of light for those struggling with cancer.

  5. Thank you–on all counts. Linnea

  6. hi there, can anyone direct me to dr shaws contact email; my father has thesame form of cancer and chemo hasnt worked,i am in greece and was wondering if we can contact directly dr. shaw and be part of a trial as in the hospital there are many patients with the same problem in the lungs.
    i would appreciate your help
    regards akis

  7. I have to say thanks for this posting… My 12 year old son had MPNST, which spread to his lungs, we were given the option to join this trial and I was put at ease reading the success stories. We’ve entered phase 6 this week, and all his bi-monthly scans have shown no new growth or tumors 🙂

    PF-02341066 for us has provided remarkable results

  8. Looking round the web, I see you are in a few blogs re: pf-02341066 linnea. Do you happen to have favorite links about the drug or discussions of it? I’d very much like them if you do and wouldn’t mind sharing.

    • Oh, and btw our trial is being held at Riley Childrens hospital of Indianapolis. We are the lucky first to try it there, not sure if IU (which is on same campus as Riley) is taking adult patients as you recommended to a lady on another blog, but since we’re receiving it in childrens ward, I’d assume chances are *better* that they’re open to offering it to adults who qualify under the trial rules also 🙂

  9. DJ’s mom–I’m so glad your son got into the trial and that the scans are coming back positive. It really has been a remarkable drug. If you’ve not checked it out yet, GRACE (http://cancergrace.org/) has several good discussions on 1066. I wish DJ the very best of luck and your family will be in my thoughts. Linnea

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